The European Commission (EC) has approved CSL’s Andembry (garadacimab) to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.
Estimated to affect one in every 50,000 people worldwide, HAE is a rare and potentially life-threatening genetic disorder characterised by recurrent and unpredictable attacks of swelling (angioedema).
These attacks are often painful and can spread to multiple parts of the body, including the abdomen, face, hands and feet, as well as the airways and gastrointestinal tract.
CSL’s Andembry is a once-monthly monoclonal antibody designed to target plasma protein factor XIIa, which plays a crucial role in initiating attacks of swelling in HAE patients.
The drug is delivered as a subcutaneous injection via a pre-filled pen and may be self-administered following appropriate training.
The EC’s decision, which comes less than a month after the Medicines and Healthcare products Regulatory Agency approved Andembry for the same patient population, was based on positive results from the phase 3 VANGUARD trial and its ongoing open-label extension study.
In VANGUARD, Andembry was shown to reduce HAE attacks rates from a mean of 2.01 attacks per month at baseline to a mean of 0.27 per month after six months of treatment.
Additionally, 61.5% of Andembry-treated patients achieved attack-free status throughout the treatment period, and the drug was shown to reduce the median number of HAE attacks to zero and lower the mean number of HAE attacks per month by 86.5% compared to placebo.
Bill Mezzanotte, executive vice president, head of research and development at CSL, said: “Andembry is a significant advancement in the management of HAE, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method.”
CSL outlined that its CSL Behring business unit will make Andembry available for eligible patients “upon completion of access and reimbursement negotiations”.
Also commenting on the approval, Henrik Balle Boysen, president of global non-profit network HAE International, said: “[Andembry], a novel once monthly subcutaneous treatment that inhibits activated factor XII, is a welcome addition to the HAE treatment landscape.
“People with HAE now have another choice for lessening the burden associated with this lifelong condition and realising the community’s shared goal of experiencing life to the fullest.”